Phase 1 Study of MM-398 Plus Cyclophosphamide in Pediatric Solid Tumors

Study ID

Cancer Related

Healthy Volunteers

Study Sites

  • Children’s Medical Center (Dallas, Plano, Southlake)

Anne Best

Principal Investigator
Patrick Leavey, M.D.

Official Title

Phase 1 Dose-escalating Study of MM-398 (Irinotecan Sucrosofate Liposome Injection) Plus Intravenous Cyclophosphamide in Recurrent or Refractory Pediatric Solid Tumors

Brief Overview

This is a Phase 1 study of the combination of two drugs: MM-398 and Cyclophosphamide. The
goal is to find the highest dose of MM-398 that can be given safely when it is used together
with the chemotherapy drug Cyclophosphamide.

Participant Eligibility

Inclusion Criteria:

- Histologically or cytologically-confirmed Ewing sarcoma, rhabdomyosarcoma,
neuroblastoma, or osteosarcoma

- Disease progression after prior therapy in locally advanced or metastatic setting

- Measurable or evaluable disease based on the Response Evaluation Criteria in Solid
Tumors (RECIST v1.1) criteria

- Age 12 months to <21 years

- Adequate bone marrow reserves, hepatic function, and renal function

- Recovered from effects of any prior surgery or cancer therapy

- Patients 18 years or older will provide written consent. A parent or legal guardian
of a patient <18 years of age will provide informed consent and patients 11 to 18
years of age will provide written assent or as per participating institutional

Exclusion Criteria:

- Clinically significant gastrointestinal disorders

- NYHA Class III or IV congestive heart failure, ventricular arrhythmias or
uncontrolled blood pressure

- Active infection or unexplained fever

- Known hypersensitivity to any of the components of MM-398 or other liposomal products

- Recent Investigational therapy

- Pregnant or breast feeding; females of child-bearing potential must test negative for
pregnancy at the time of enrollment